The Committee for Advanced Therapies is the European Medicines Agency’s specialized scientific body for Advanced Therapy Medicinal Products. It prepares a draft opinion on quality, safety, and efficacy for an ATMP marketing authorisation application. Its work prepares the opinion of the Committee for Medicinal Products for Human Use, but does not replace it.
Mandate under the ATMP Regulation
Regulation (EC) No 1394/2007 established the Committee for Advanced Therapies, or CAT, within the EMA. Its specific task is to formulate a draft opinion for each ATMP marketing authorisation application submitted to the Agency. For this purpose, the Committee pools expertise on gene therapy, somatic cell therapy, tissue engineering, and combined ATMPs.
The CAT can also, at the request of the EMA Executive Director or the European Commission, issue opinions on scientific questions related to ATMPs. It participates in the classification of products and the certification procedure for SMEs. Its mandate is thus broader than individual marketing authorisation assessment, but remains focused on Advanced Therapy Medicinal Products.
From Draft to EMA Opinion
According to the EMA’s presentation, the CAT submits its draft before the Committee for Medicinal Products for Human Use, or CHMP, adopts the final opinion on the marketing authorisation. The CAT is therefore not the body that adopts the final positive or negative CHMP opinion. This institutional sequence is essential for attributing scientific work and formal decision-making.
The European Commission then decides on the marketing authorisation in the centralised procedure. Therefore, in a communication on the development program, a clear distinction should be made between CAT assessment, CHMP opinion, and Commission decision. The designation “EMA authorisation” shortens this multi-stage process and does not describe a competence of the CAT alone.
Distinction from the CHMP and other stakeholders
The Committee for Advanced Therapies is a preparatory ATMP body; the final scientific opinion on a marketing authorisation application is adopted by the CHMP. The CAT provides the specialized draft for this. This is different from the decision of the European Commission, which grants or refuses the marketing authorisation.
The existing entry food-and-drug-administration refers to a US authority and not a European committee structure. The International Council for Harmonisation develops international guidelines but does not conduct ATMP application assessments. The PRAC also has a different mandate: its focus is on pharmacovigilance rather than the ATMP draft opinion.
The composition of the CAT is tailored to the specialized field. In addition to members from the Member States, experts with scientific experience in the relevant areas may be involved, according to the rules of the ATMP Regulation. This ensures that the assessment is not solely guided by general medicinal product issues, but also by the specific characteristics of living cells, tissues, recombinant nucleic acids, and integrated medical devices. For applicants, this does not result in a separate authorisation route alongside the centralised procedure. However, it explains why the scientific evaluation of an ATMP is organised via the CAT at a preparatory stage and which technical requirements should be proactively addressed during development.
For classification, the CAT can be involved early with regard to product description and mechanism of action. A classification recommendation does not create market authorisation and is not equivalent to the certification procedure for SMEs. It answers the fundamental question of whether a specific product meets the ATMP definition. The certification procedure, on the other hand, evaluates specific data packages. These separate tasks explain why the CAT can be involved in both processes without their results being interchangeable.
The CAT’s Rules of Procedure form the formal framework for meetings, reporting, and decision-making. They supplement the legal mandate without altering the CHMP’s responsibility for the final opinion. For development planning, the CAT’s scientific specialisation is therefore relevant, but procedural authority remains clearly distributed within the EMA system.
Relevance for clinical trials
The subsequent CAT assessment already influences during clinical development which product characterisation, non-clinical justifications, and clinical data should be coherently prepared. For an ATMP, it must be comprehensible how the product’s properties are linked to dosage, administration, safety monitoring, and efficacy endpoints. A fragmented presentation between CMC and clinical documents complicates the overall assessment.
Full-service CROs like Mediconomics support ATMP studies through regulatory development planning and consistent preparation of the study protocol, Investigator’s Brochure, and clinical summaries. They coordinate medical writing, data management, and project management with product-related expert contributions to ensure that robust, version-controlled evidence is available for subsequent CAT and CHMP procedures.
Frequently Asked Questions (FAQ)
Does the CAT adopt the final marketing authorisation opinion?
No. The CAT prepares the draft for the ATMP application. The final opinion on the marketing authorisation is adopted by the CHMP.
Can the CAT classify products before a marketing authorisation application is submitted?
Yes. The EMA names the scientific recommendation for ATMP classification as a task that can help developers determine a product’s regulatory status.
Is the CAT responsible for all medicinal products for human use?
No. Its specific legal mandate concerns Advanced Therapy Medicinal Products. For other medicinal products, the respective procedures and EMA bodies apply.
Regulatory References
- Regulation (EC) No 1394/2007 on Advanced Therapy Medicinal Products – establishes the CAT and defines its scope of duties.
- EMA Committee for Advanced Therapies (CAT) – describes drafting, classification, and certification in practice.
- EMA CAT Rules of Procedure – regulates the working methods of the Committee.